Treatment of retinitis pigmentosa

Abstract

A polynucleotide comprising a nucleotide sequence encoding the retinitis pigmentosa GTPase regulator ORF15 isoform (RPGRORF15), wherein the RPGRORF15-encoding nucleotide sequence has been codon optimised to increase fidelity of replication of the sequence.

What is claimed is: 
     
       1. A composition comprising a nucleic acid comprising
 (a) a sequence encoding a rhodopsin kinase promoter, and 
 (b) a sequence encoding a retinitis pigmentosa GTPase regulator ORF15 isoform (RPGR ORF15 ) and comprising a nucleotide sequence that has at least 80% identity to the nucleic acid sequence of SEQ ID NO: 3. 
 
     
     
       2. The composition of  claim 1 , wherein the sequence encoding the promoter has at least 80% identity to the nucleic acid sequence of SEQ ID NO: 7. 
     
     
       3. The composition of  claim 1 , wherein the sequence encoding the promoter has at least 90% identity to the nucleic acid sequence of SEQ ID NO: 7. 
     
     
       4. The composition of  claim 1 , wherein the sequence encoding the promoter comprises the nucleic acid sequence of SEQ ID NO: 7. 
     
     
       5. The composition of  claim 1 , wherein the sequence encoding the promoter consists of the nucleic acid sequence of SEQ ID NO: 7. 
     
     
       6. The composition of  claim 1 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
       7. The composition of  claim 2 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
       8. The composition of  claim 3 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
       9. The composition of  claim 4 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
       10. The composition of  claim 5 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence encoding an amino acid sequence that has at least 80% identity to the amino acid sequence of SEQ ID NO: 1. 
     
     
       11. The composition of  claim 1 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3. 
     
     
       12. The composition of  claim 2 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3. 
     
     
       13. The composition of  claim 3 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3. 
     
     
       14. The composition of  claim 4 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3. 
     
     
       15. The composition of  claim 5 , wherein the sequence encoding the RPGR ORF15  comprises a nucleotide sequence that has at least 97% identity to the nucleic acid sequence of SEQ ID NO: 3.